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INTRODUCTION

Regenerative medicine is a complex and rapidly advancing field that holds tremendous promise in treating, and even curing, many diseases. The understanding and control of tissue repair is one of the most urgent challenges in medicine today. Regenerative medicine seeks to either recruit the patient’s reparative cells or to replace the malfunctioning tissue altogether to restore the deficient organ to adequate function. The common link among all types of regenerative therapies is the stem cell, which gives all tissues the capacity to regenerate. The mechanisms underlying the ability of a progenitor cell to differentiate have been challenging to elucidate, with recent experimentation focused on editing the genome itself. It has been even more difficult to determine how a differentiated cell can be instructed to revert to an immature state and undergo a re-specification to another differentiated cellular phenotype or an asymmetrical division to generate more immature cells. Our ability to modify genomes, harness stem cells, and transplant autologous or allogeneic tissues has transformed biomedical inquiry and offers hope to patients with diseases spanning all organ systems, including cardiac, lung, central nervous system, and liver and pancreatic diseases.

Regenerative medicine is a concept that evolved from knowledge in genome regulation and modification, from understanding of embryonic development and “stemness” of cells, and from 50 years of experience in human transplant biology. Therefore, a narrow view of any of these disciplines is not sufficient for illuminating the mechanisms of action underlying the already accomplished successes and for guiding the potential of novel basic biology discoveries into clinically meaningful regenerative medicine (Fig. 30–1).

Figure 30–1.

The three-body problem of regenerative medicine. The three factors—cell, genome, and patient—influence each other in complex and sometimes unexpected ways. These three separate scientific foci of regenerative medicine must be developed in the context of one another to have meaningful impact.